First, let's talk about what we mean by on "label" drugs. In the U. S. , the FDA requires that drug makers submit, test and get approval from the FDA for specific uses of their product before the product can ever be marketed. To obtain approval, pharmaceutical manufacturers must submit extensive study data demonstrating safety and effectiveness, a process which usually takes several years and millions of dollars. When a product is approved by the FDA, a specific "label" for that product is approved too. The label must include the product's approved indications, dosage and method of administration, and use in specific populations.

So, what is "off-label"? After the FDA approves a drug for its specific use, physicians can prescribe the product for uses not covered by the approved label. This is "off-label" use. Off-label uses include:a drug prescribed to patients with conditions not listed on the approved label; departure from the approved drug dosage, method of administration, or patient population; and unapproved product combinations, such as using two different drugs to treat a single condition. This practice is perfectly legal but it lacks any kind of oversight.

"Off-label" drugs for back pain. Among the types of drugs commonly prescribed off-label, most common were heart drugs and anti-convulsants (indicated for seizures). For example, gabapentin, brand name Neurontin, had one of the highest proportions of off-label use among specific medications, namely for neuropathic pain (it is only indicated for use in controlling epileptic seizures and in pain from post-herpetic neuralgia). Neurontin also happens to be a quite commonly prescribed drug for people diagnosed with chronic back pain or chronic low back pain. This of course means the drug is being prescribed off-label in the back pain patient population.

Patients should always consider carefully all medications that are prescribed to them. It is fair for patients to ask their doctors whether a prescription is on or off-label, and this can be easily and more thoroughly researched on reliable health information websites that address medications, such as MedlinePlus, the FDA, or condition-specific sites. If off-label, it is important to know whether there is sufficient data to support its use or if the practitioner's decision is based on anecdotal evidence. The patient should weigh off-label recommendations against the alternatives with his/her practitioner, and ultimately decide if he/she is comfortable with an off-label drug for the specific health issue being addressed. It is important for patients to keep in mind that a drug prescribed off-label to them is a drug that has not been approved by the FDA for some element of their specific health situation.

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Is "off-label" drug prescribing good or bad? There isn't a clear cut answer. Anecdotally, many of my colleagues and personal circle of friends have experienced off-label drug use with positive results. For example, our children have been prescribed albuterol for various severe pulmonary episodes (not asthma, for which it's indicated) with much success. The Spine-health.com message boards are full of stories of people taking medications off-label for back pain or leg pain as prescribed by their doctors, with many experiencing much needed pain relief not possible with approved drugs. So, off-label use can be positive in that it expands access to important health benefits that may not be possible with alternatives. On the flip side, concerns and warning bells should go off with respect to safety and the long-term impact of a drug use that has not been extensively studied.

The study authors cited several reasons as to why the high percentage of off-label prescribing is occurring, with some reasons being legitimate and others not. The practice itself raises issues that require addressing, including a better/faster process for getting legitimate additional uses for a drug indicated, but also for ensuring patient safety for patients dutifully following an off-label treatment protocol as prescribed by physicians they trust.

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